Albireo Pharma, Inc. (Nasdaq: ALBO), a rare liver disease
company developing novel bile acid modulators to treat pediatric
and adult liver diseases, today announced that the Italian
Medicines Agency, also known as Agenzia Italiana del Farmaco
(AIFA), has enabled reimbursed access to Bylvay (odevixibat) for
the treatment of all types of progressive familial intrahepatic
cholestasis (PFIC) patients in Italy. A potent, oral, once-daily,
non-systemic ileal bile acid transport inhibitor, Bylvay has
minimal systemic exposure and acts locally in the small intestine
as a treatment for PFIC, a rare and devastating disorder that
causes progressive, liver disease.
"It’s extremely important to see the positive decision by AIFA
to enable access to Bylvay to treat patients in need. As the first
approved medicine for PFIC, I’m relieved to now be able to offer a
non-surgical treatment option that provides meaningful clinical
benefits that can improve the lives of patients and families,” said
Dr. Lorenzo D’Antiga, Director, Unit of Hepatology,
Gastroenterology and Pediatric Transplantation, Papa Giovanni XXIII
Hospital.
Patients with PFIC have impaired bile flow, or cholestasis, and
the resulting bile build-up in liver cells causes life-threatening
liver disease and symptoms, such as intense itching, poor sleep,
delayed growth, and diminished quality of life. The harmful impacts
of the disease extend beyond the individuals with PFIC to those
caring for them as shown by the 2022 multinational PICTURE study,
which revealed PFIC negatively affects caregivers’ quality of life,
relationships, and career prospects.
“The burden of the disease is devastating for children and
families living with PFIC. Severe itching can cause a tremendous
impact on patients and disrupt basic daily activities resulting in
loss of sleep, poor attention, and lower school performance.
Intractable pruritus can, by itself, justify liver transplantation
in these patients. We are thrilled to have Bylvay as an innovative
therapeutic non-surgical option that could significantly improve
disease management and, potentially, modify the natural history of
the disease,” said Prof. Giuseppe Maggiore, Director of the
Division of Hepatology, Gastroenterology, Digestive Endoscopy, and
Nutrition, Pediatric Hospital Bambino Gesù.
Bylvay was granted “Full Therapeutic Innovation” status by AIFA,
a special designation applied to select drug applications that
address a high unmet need, added clinical benefit, and robustness
of the scientific evidence. This status provides access to
dedicated national funding. For Healthcare Provider General
Inquires, please email: Italy@albireopharma.com
“Bylvay has the potential to transform the lives of those
impacted by PFIC, and availability in Italy marks an important
milestone for our PFIC community,” explained Francesca Lombardozzi,
Head of PFIC Italia Network and the mother of a 3-year-old child
living with PFIC. “I know firsthand the extreme burden and
challenges PFIC patients and caregivers are facing. This is a great
first step for the whole community, and we hope others will follow.
I’m grateful for the Albireo team’s continued efforts to provide
access to this critical medication that will make a huge impact on
our lives.”
“We are grateful to AIFA for this positive decision, recognizing
the clinical and economic value of Bylvay,” said Thomas Topini,
General Manager, Italy at Albireo. “It is because of AIFA’s
engagement that we were able to accelerate reimbursed access to
Bylvay and help PFIC patients and families in Italy living with the
devastating effects of this disease.”
Bylvay was approved in Europe for the treatment of all types of
PFIC in patients aged 6 months or older, based on the PEDFIC 1
study and PEDFIC 2 open-label extension (OLE) study. This
availability marks the fourth reimbursement approval for Bylvay in
Europe, following launches in Germany, England, Wales &
Northern Ireland, and Scotland. Bylvay is currently being evaluated
in the ASSERT Phase 3 study for Alagille syndrome (ALGS) and in the
BOLD Phase 3 study for patients with biliary atresia. The ASSERT
and BOLD studies remain on track to report topline data in the fall
of 2022 and 2024, respectively.
PEDFIC 1 & PEDFIC 2 Studies
The decision by the Italian Medicines Agency is based on data
from PEDFIC 1 and PEDFIC 2, the largest, global, Phase 3 trials
ever conducted in PFIC. In PEDFIC 1, a randomized, double-blind,
placebo-controlled study, Bylvay met both its pruritus (p=0.004)
and serum bile acid (p=0.003) primary endpoints and was well
tolerated with low incidence of drug-related diarrhea/frequent
bowel movements (9.5% of treated patients vs. 5.0% of placebo
patients). PEDFIC 2, a long-term, open-label Phase 3 extension
study, affirmed Bylvay delivered sustained reductions in serum bile
acid, as well as improvements in pruritus assessments, growth and
sleep and markers of liver function in patients treated up to 48
weeks in an interim analysis. Across both studies, Bylvay was well
tolerated. The most common adverse reactions for Bylvay were
diarrhea, liver test abnormalities, vomiting, abdominal pain, and
fat-soluble vitamin deficiency. There were no serious
treatment-related adverse events reported in any clinical study
with Bylvay.
About Bylvay (odevixibat)
Bylvay is the first drug approved in the U.S. for the treatment
of pruritus in patients 3 months of age and older in all types of
progressive familial intrahepatic cholestasis (PFIC). Limitation of
Use: Bylvay may not be effective in PFIC type 2 patients with
ABCB11 variants resulting in non-functional or complete absence of
bile salt export pump protein (BSEP-3). The European Commission
(EC) and UK Medicines and Healthcare Products Regulatory Agency
(MHRA) have also granted marketing authorization of Bylvay for the
treatment of PFIC in patients aged 6 months or older. Bylvay is
available in Italy, Germany and the UK and will be available for
sale in other European countries following pricing and
reimbursement approval. A potent, once-daily, non-systemic ileal
bile acid transport inhibitor, Bylvay acts locally in the small
intestine. Bylvay can be taken as a capsule for patients that are
able to swallow capsules, or opened and sprinkled onto food, which
is a factor of key importance for adherence in a pediatric patient
population. The most common adverse reactions for Bylvay are
diarrhea, liver test abnormalities, vomiting, abdominal pain, and
fat-soluble vitamin deficiency. The medicine can only be obtained
with a prescription. For more information about using Bylvay, see
the package leaflet or contact your doctor or pharmacist. For full
prescribing information, visit www.bylvay.com.
In the U.S. and Europe, Bylvay has orphan exclusivity for its
approved PFIC indications, and orphan designations for the
treatment of ALGS, biliary atresia and primary biliary cholangitis.
Bylvay is being evaluated in the ongoing PEDFIC 2 open-label trial
in patients with PFIC, in the BOLD Phase 3 study for patients with
biliary atresia and the ASSERT Phase 3 study for ALGS.
Important Safety Information
- The most common adverse reactions
for Bylvay are diarrhea, liver test abnormalities, vomiting,
abdominal pain, and fat-soluble vitamin deficiency.
- Liver Test
Abnormalities: Patients should obtain baseline liver tests and
monitor during treatment. Dose reduction or treatment interruption
may be required if abnormalities occur. For persistent or recurrent
liver test abnormalities, consider treatment discontinuation.
- Diarrhea: Treat
dehydration. Treatment interruption or discontinuation may be
required for persistent diarrhea.
- Fat-Soluble Vitamin
(FSV) Deficiency: Patient should obtain baseline vitamin levels and
monitor during treatment. Supplement if deficiency is observed. If
FSV deficiency persists or worsens despite FSV supplementation,
discontinue treatment.
About Albireo
Albireo Pharma is a rare disease company focused on the
development of novel bile acid modulators to treat pediatric and
adult liver diseases. Albireo’s lead product, Bylvay, was approved
by the U.S. FDA as the first drug for the treatment of pruritus in
all types of progressive familial intrahepatic cholestasis (PFIC),
and it is also being developed to treat other rare pediatric
cholestatic liver diseases with Phase 3 trials in Alagille syndrome
(ALGS) and biliary atresia, as well as Open-label Extension (OLE)
studies for PFIC and ALGS. In Europe, Bylvay is reimbursed for the
treatment of PFIC in Germany, England, Wales & Northern
Ireland, Scotland, and Italy. The Company has also completed a
Phase 1 clinical trial for A3907 to advance development in adult
cholestatic liver disease, with IND-enabling studies progressing
with A2342 for viral and cholestatic liver disease. Albireo was
spun out from AstraZeneca in 2008 and is headquartered in Boston,
Massachusetts, with its key operating subsidiary in Gothenburg,
Sweden. For more information on Albireo, please visit
www.albireopharma.com.
Forward-Looking Statements
This press release includes “forward-looking statements” within
the meaning of the Private Securities Litigation Reform Act of
1995. Forward-looking statements include statements, other than
statements of historical fact, regarding, among other things:
Albireo’s commercialization plans; the plans for, or progress,
scope, cost, initiation, duration, enrollment, results or timing
for availability of results of, development of Bylvay, A3907, A2342
or any other Albireo product candidate or program; the PEDFIC 2
open-label trial in patients with PFIC; the pivotal trial for
Bylvay in biliary atresia (BOLD); the pivotal trial for Bylvay in
Alagille syndrome (ASSERT); the Phase 2 study for A3907 the
IND-enabling or clinical studies for A2342; the target
indication(s) for development or approval; the timing for
initiation or completion of or availability or reporting of results
from any clinical trial, including the long-term open-label
extension study for Bylvay in PFIC, the BOLD and ASSERT trials, the
Phase 2 study for A3907, and the IND-enabling and clinical studies
for A2342; potential regulatory approval and plans for potential
commercialization of Bylvay in additional countries; the potential
benefits or competitive position of Bylvay or any other Albireo
product candidate or program or the commercial opportunity in any
target indication;; or Albireo’s plans, expectations or future
operations, financial position, revenues, costs or expenses.
Albireo often uses words such as “anticipates,” “believes,”
“plans,” “expects,” “projects,” “future,” “intends,” “may,” “will,”
“should,” “could,” “estimates,” “predicts,” “potential,” “planned,”
“continue,” “guidance,” or the negative of these terms or other
similar expressions to identify forward-looking statements. Actual
results, performance or experience may differ materially from those
expressed or implied by any forward-looking statement as a result
of various risks, uncertainties and other factors, including, but
not limited to: results achieved in Bylvay in the treatment of
patients with PFIC may be different than observed in clinical
trials, and may vary among patients; potential negative impacts of
the COVID-19 pandemic, including on manufacturing, supply, conduct
or initiation of clinical trials, or other aspects of our business;
whether favorable findings from clinical trials of Bylvay to date,
including findings in indications other than PFIC, will be
predictive of results from other clinical trials of Bylvay; the
timing for initiation or completion of, or for availability of data
from, clinical trials of Bylvay, including BOLD and ASSERT and the
Phase 2 clinical trial of A3907, and the outcomes of such trials;
Albireo’s ability to obtain coverage, pricing or reimbursement for
approved products in the United States or Europe; delays or other
challenges in the recruitment of patients for, or the conduct of,
the Company’s clinical trials; and the Company’s critical
accounting policies. These and other risks and uncertainties that
Albireo faces are described in greater detail under the heading
“Risk Factors” in Albireo’s most recent Annual Report on Form 10-K
or in subsequent filings that it makes with the Securities and
Exchange Commission. As a result of risks and uncertainties that
Albireo faces, the results or events indicated by any
forward-looking statement may not occur. Albireo cautions you not
to place undue reliance on any forward-looking statement. In
addition, any forward-looking statement in this press release
represents Albireo’s views only as of the date of this press
release and should not be relied upon as representing its views as
of any subsequent date. Albireo disclaims any obligation to update
any forward-looking statement except as required by applicable
law.
Media Contacts:
Colleen Alabiso, 857-356-3905,
colleen.alabiso@albireopharma.com Lance Buckley,
917-439-2241, lbuckley@lippetaylor.com
Investor Contact:Hans Vitzthum, LifeSci
Advisors, LLC., 617-430-7578
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